KR102630217B1 - 혈색소병증의 치료를 위한 조성물 및 방법 - Google Patents

혈색소병증의 치료를 위한 조성물 및 방법 Download PDF

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KR102630217B1
KR102630217B1 KR1020197026089A KR20197026089A KR102630217B1 KR 102630217 B1 KR102630217 B1 KR 102630217B1 KR 1020197026089 A KR1020197026089 A KR 1020197026089A KR 20197026089 A KR20197026089 A KR 20197026089A KR 102630217 B1 KR102630217 B1 KR 102630217B1
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cells
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molecule
grna
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크레이그 스티븐 미카닌
크리스티안 슈메트
제니퍼 스니드
수잔 씨. 스티븐슨
이 양
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노파르티스 아게
인텔리아 테라퓨틱스, 인크.
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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KR1020197026089A 2017-02-06 2018-02-05 혈색소병증의 치료를 위한 조성물 및 방법 Active KR102630217B1 (ko)

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KR1020247002710A KR20240017098A (ko) 2017-02-06 2018-02-05 혈색소병증의 치료를 위한 조성물 및 방법

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US201762455464P 2017-02-06 2017-02-06
US62/455,464 2017-02-06
PCT/IB2018/050712 WO2018142364A1 (en) 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies

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KR102630217B1 true KR102630217B1 (ko) 2024-01-29

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KR1020247002710A Pending KR20240017098A (ko) 2017-02-06 2018-02-05 혈색소병증의 치료를 위한 조성물 및 방법

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US (2) US11466271B2 (enExample)
EP (1) EP3577223A1 (enExample)
JP (3) JP2020505934A (enExample)
KR (2) KR102630217B1 (enExample)
CN (2) CN110546262B (enExample)
AR (1) AR110962A1 (enExample)
AU (3) AU2018215726B2 (enExample)
BR (1) BR112019016205A2 (enExample)
CA (1) CA3052275A1 (enExample)
EA (1) EA201991862A1 (enExample)
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PH (1) PH12019501812A1 (enExample)
SA (1) SA519402400B1 (enExample)
SG (1) SG11201907056XA (enExample)
TW (1) TW201839136A (enExample)
WO (1) WO2018142364A1 (enExample)

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